Abstract
Over the past decade, research into circular RNA (circRNA) has increased rapidly, and over the past few years, circRNA has emerged as a promising therapeutic platform. The regulatory functions of circRNAs, including their roles in templating protein translation and regulating protein and RNA functions, as well as their unique characteristics, such as increased stability and a favourable immunological profile compared with mRNAs, make them attractive candidates for RNA-based therapies. Here, we describe the properties of circRNAs, their therapeutic potential and technologies for their synthesis. We also discuss the prospects and challenges to be overcome to unlock the full potential of circRNAs as drugs.
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Acknowledgements
This work was funded by a grant from the Danish National Research Foundation to the Centre for Cellular Signal Patterns (CellPAT) (grant DNRF 135) and by the Novo Nordisk Foundation (grant NNF23OC0081177). The authors thank M. Gockert for proofreading the manuscript and for valuable comments.
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E.O’L. and T.B.H. are employees of Circio AB, which is developing vector-based circular RNA therapeutics. The other authors declare no competing interests.
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O’Leary, E., Jiang, Y., Kristensen, L.S. et al. The therapeutic potential of circular RNAs. Nat Rev Genet 26, 230–244 (2025). https://doi.org/10.1038/s41576-024-00806-x
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DOI: https://doi.org/10.1038/s41576-024-00806-x
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